Medical and Regulatory Affairs

Medical and Regulatory Affairs

Long-Term Gene Therapy Follow-Up: Patient Considerations

When developing a long-term follow-up strategy for gene therapy trials, safety is the main concern, closely followed by the patient perspective. Digital technologies and monitoring have proven essential to this, easing the burden on the patient, and providing accurate, time-saving methods of data management. Momentum is increasing in the field of gene therapy, with over...

Medical and Regulatory Affairs

How COVID-19 Is Making Operationalizing Gene Therapy Trials Even Tougher – And How We Handled It

Though gene therapy has been around for decades, it continues to pose extraordinary challenges in the areas of R&D, clinical development, and operation of clinical trials. Sponsors and CROs face shifting operational and regulatory demands amid rapid advances in the science of precision medicine. In our previous blog post, we discussed site selection, manufacturing, long-term...

Medical and Regulatory Affairs

Site Selection, Manufacturing & Long-Term Follow-Up: Looming Challenges for a Rescue Phase 1/2 Gene Therapy Trial

Designing and conducting a gene therapy trial is a complex undertaking. Understanding, planning for, and overcoming the myriad challenges of operationalizing these studies will help you bring safe, breakthrough treatments to patients with unmet medical needs. In this blog post, we introduce a case study as a framework for exploring critical study design considerations of...

Consulting

How to Navigate Global Regulatory Frameworks & Expedited Programs for Gene Therapy Development in the U.S., EU, & Japan

As scientific knowledge, clinical experience, and acceptance of gene therapy products have evolved, so have the regulatory frameworks for ensuring the safety of these novel treatments. To date, there is no harmonized international standard for regulating gene therapy products; however, the U.S., EU, and Japan have established regulatory frameworks with subtle variations. Understanding how gene...

Clinical Research: Phase 1 - Phase 4

Choose the Right FDA Program to Expedite Your Rare Oncology Trial

Rare cancers account for 27 percent of all new cancer diagnoses in the U.S. and 22 percent of all new cancer diagnoses in the EU.1 With their poorly understood natural histories, phenotypic heterogeneity, and diverse clinical manifestations, rare cancers pose challenges to drug development and represent a significant unmet need in oncology. Faced with limited treatment...

Medical and Regulatory Affairs

Dermatology Webinar Series: Looking Ahead to the Future of Dermatology Research

New treatment options for moderate to severe dermatological diseases and areas of high unmet need are driving a revolution in clinical development of dermatology drugs. But questions remain: What indications should be pursued? How to design a study that targets the right endpoints? And how will environmental constraints affect the research? In this three-part series,...

Clinical Research: Phase 1 - Phase 4

Drug and Device Development Secrets in the World of Medical Aesthetics

New treatment options for aesthetic indications are in record-high demand, fueled by an aging population, and a growing focus on aesthetic issues related to nutritional habits, sedentary lifestyles, the complex stresses of 21st-century life and rising awareness about aesthetic solutions in all adult age groups. The search for more effective, less invasive, more affordable and safer solutions...

Consulting

Dec. 8: Virtual Presentation – Effects of Digitization on Patients at the Center of the Clinical Study

8 December 2020 | 6:30pm CET Effects of Digitization on Patients at the Center of a Clinical Study: Regulatory Aspects, Initiatives And ExamplesPresented by Industrial Pharmaceuticals Division, German Pharmaceutical Society

Consulting

Beware of Underestimating the Value of a Pre-NDA Meeting

Drug development is a resource-intensive endeavor. Seeking input from the U.S. Food and Drug Administration throughout the journey can help optimize those resources and maximize the likelihood of regulatory approval. When preparing to submit a new drug application (NDA), a pre-NDA meeting with the FDA can be a critical step in ensuring the submission of...

Clinical Research: Phase 1 - Phase 4

Adaptive Design Methods Offer Rapid, Seamless Transition Between Studies

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers , each with a unique implication for patient management and therapy....